CRISPR TherapeuticsIn Development
A next-generation, deviceless beta-cell replacement candidate for type 1 diabetes, made of unencapsulated precursor islet cells derived from gene-edited, hypoimmune induced pluripotent stem cells (iPSCs), designed for direct administration without an encapsulation device or chronic immunosuppression.
Latest update Aug 17, 2026
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Preclinical. Succeeds the earlier CTX211 (formerly VCTX211/VCTX210) program, whose Phase 1 data showed detectable C-peptide 12 months after implantation; that proof-of-concept informed CTX213's hypoimmune engineering. CTX213 has shown compelling preclinical efficacy via direct administration and is progressing toward the clinic, with no IND filed yet as of Q2 2026.
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The world's first approved CRISPR/Cas9 gene-editing medicine (exagamglogene autotemcel). A one-time therapy that edits the BCL11A gene in a patient's own blood stem cells to reactivate fetal hemoglobin, treating sickle cell disease and transfusion-dependent beta thalassemia. Partnered with Vertex Pharmaceuticals.
An in vivo gene-editing therapy delivered by lipid nanoparticles that knocks out the ANGPTL3 gene in the liver to lower triglycerides and LDL cholesterol - a potential one-time treatment for severe dyslipidemia and cardiovascular risk, and the lead of CRISPR's in vivo pipeline.
A long-acting, double-stranded siRNA that targets Factor XI (FXI) messenger RNA to inhibit FXI protein expression, aiming to provide anticoagulant and antithrombotic protection with less bleeding risk than existing anticoagulants, potentially dosed as infrequently as semi-annually. Co-developed with Sirius Therapeutics (50/50 cost and profit share; CRISPR leads U.S. commercialization, Sirius leads Greater China) across a range of thromboembolic indications including atrial fibrillation, venous thromboembolism, and stroke prevention.
An allogeneic, gene-edited anti-CD19 CAR-T cell therapy (zugocaptagene geleucel) being developed for autoimmune diseases such as lupus as well as relapsed/refractory B-cell cancers, using CRISPR editing to make an off-the-shelf product.
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Oklo
Oklo's fast-fission powerhouse product line is designed for build-own-operate deployment, targeting customers that want firm clean power in a smaller footprint than traditional large reactors.
Oklo
Oklo is developing an advanced fuel center to recycle used nuclear fuel and fabricate metal fuel for Aurora and other fast-reactor applications.
TerraPower
Sodium-cooled fast reactor with integrated molten salt energy storage, enabling flexible output from 345 MWe to 500 MWe peak.
TerraPower
Integrated thermal energy storage system using molten salt to decouple reactor output from grid delivery, enabling peak power output.